Small patient populations: Rare diseases have small, dispersed patient populations, restricting the potential for large randomised clinical trials. In some cases, companies need to seek a licence for a product in countries where they have not been able to trial it on patients. This can present serious longer-term challenges, with reimbursement bodies often basing their decisions on data from large Phase III studies and looking for local data to support their assessment.
Limited clinical knowledge and pathways: In rare disease communities there’s often limited clinical knowledge about new drugs, making it difficult to benchmark standards of care or existing patient pathways. Sometimes there may not be a pathway at all. Likewise, the expert community will often be tiny; in some countries, particularly those where trials have not taken place, companies can struggle to identify clinical experts to champion innovation.
High price: The challenges of clinical development mean it can be expensive to bring a rare cancer therapy to market. This is often reflected in high prices as companies strive for a commercial return. The subject of drug pricing has long been debated with pharma working hard to shift the emphasis from ‘cost’ to ‘value’ – but it’s much harder in rare diseases.
Limited evidence: Traditional methods of gathering evidence are seldom available in rare cancers. Active comparators are often rare or non-existent, while both safety and long-term monitoring data can be insufficient. Similarly, selecting the right surrogate endpoint can be problematic. These factors make it hard to develop the evidence to convince payers, healthcare professionals and patients that a drug is worth using. To combat this, policies to support early access to medicines have emerged. However, these schemes – which allow medicines to be used prior to marketing authorisation subject to the mandatory capture of clinical and cost-effectiveness data in the real world – are not without risk. The example of a pharma company withdrawing a rare sarcoma treatment two and a half years after it gained accelerated approval highlights the challenges of fast-tracking access on limited evidence.
Planning, and then executing those plans early is key to getting innovations in rare cancers to patients safely and quickly. It’s important to be proactive in considering the evidence required to gain reimbursement and access. This means engaging early with all stakeholders to understand what value means to them and the data needed to demonstrate it. Early engagement is as important internally as it is with customers and users. The different objectives of clinical trial groups and market access teams may hinder a company’s ability to identify, co-ordinate and collect data that will either help pricing and reimbursement or demonstrate value to clinicians and patients. With clinical trial programmes in rare oncology naturally lean, it’s important to establish – from the very beginning – the endpoints that are likely to resonate with your key stakeholder groups. This will only come through proactive (and ongoing) engagement with external influencers and joined-up thinking across the organisation. Direct engagement with patients or through patient advocacy groups (PAGs) at this early stage provides an invaluable opportunity to incorporate the patient voice.
Agility is crucial. The healthcare environment is dynamic, so companies must be able to react quickly to what they learn. Preparation is key; it’s important to understand current clinical practice and policy levers that could create opportunities for your intervention. Accelerated access schemes or managed access agreements provide a mechanism to capture real-world evidence that could secure access, but without proper planning, companies can sometimes be wrong-footed when marketing authorisation is expedited and squander the opportunity to gather the best evidence.
With good, proactive engagement, pharma has an opportunity to partner with payers (and indeed patients) to co-create evidence-based pricing and contracting models that support value-based healthcare. There’s a growing willingness for partnership, but it’s for pharma to broker discussions with ideas on what those new models might look like.
With patient populations in rare cancers extremely small, companies know that every patient in a study is gold dust; they can’t afford to lose anyone to follow-up. There’s increasing investment in supporting patients through clinical trials – using a variety of technologies and engagement methods – to ensure everyone recruited to a study stays on it. However, engagement doesn’t end at the clinical trial; companies also need to consider long-term follow-up and how they sustain patient engagement in the real world. Innovation is critical. Successful companies seek active involvement of patients as experts by experience through building strong, long and collaborative relationships with PAGs to ensure the patient voice is present and influential at every step of the pathway.
Amanda Pulfer, strategic director, OPEN VIE
Amanda has worked in the real-world evidence arena for more than 10 years offering strategic consultancy to support clients in the development and delivery of real-world evidence programmes. Prior to consulting, Amanda worked in pharma in a number of commercial roles with responsibility for launch and mature brands. More recently she has become involved in the growing area of patient engagement supporting clients to ‘measure what matters’ to patients in real-world studies and clinical trials and embedding the active involvement of patients and PAGs as experts by experience along the journey to successful reimbursement.
Cathy Wright, director of market access, OPEN VIE
After starting her career in the pharma industry, Cathy has worked extensively in strategic market access consultancy, delivering a wide range of market access programmes and developing value strategies; ensuring their effective communication and dissemination to all stakeholders. Cathy has particular experience in hospital specialist and oncology market access and has worked across many early access projects including the Promising Innovative Medicine (PIM) and Early Access to Medicines Scheme (EAMS) submissions as well as HTA submissions to NICE and SMC.